Organoid-based Sensitivity-guided Chemotherapy for Advanced / Refractory Pediatric Tumors

Официальное название

A Clinical Study on Organoid-Based Drug Sensitivity-Guided Chemotherapy for High-Risk, Relapsed, and Refractory Pediatric Soft Tissue Malignant Tumors

Обзор

This is a prospective, open-label, single-arm, single-center study designed to evaluate the feasibility of using patient-derived organoid drug sensitivity analysis to guide chemotherapy for pediatric patients with refractory and relapsed malignant soft tissue tumors. The primary endpoint of the study is objective response rate (ORR), and the secondary endpoint is event-free survival (EFS).

Подробное описание

This is a prospective, open-label, single-arm, single-center study with a planned total enrollment of 30 pediatric patients with refractory and relapsed malignant soft tissue tumors. Patients will be enrolled in this clinical trial after providing informed consent and meeting the inclusion and exclusion criteria. As this is a single-arm study without a control group, all patients will be assigned to the experimental arm. Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response. Clinical efficacy will be assessed during follow-up to evaluate the feasibility of using organoid drug sensitivity analysis to guide chemotherapy for refractory and relapsed malignant soft tissue tumors in children.

Вмешательства

• Препарат treatment based on the results of drug sensitivity analysis
  Treatment regimens will be comprehensively evaluated and adjusted based on the results of patient-derived organoid drug sensitivity testing in combination with clinical response.

Первичные конечные точки

• objective response rate (ORR) [Срок оценки: from the first drug administration up to two years]

Вторичные конечные точки (1)

• Event-Free Survival (EFS) [Срок оценки: from the first drug administration up to two years]

Критерии участия

Критерии включения

• Age between 1 and 18 years, gender unrestricted.
• Patients with histopathologically confirmed rhabdomyosarcoma, Ewing sarcoma, malignant rhabdoid tumor, or non-rhabdomyosarcoma soft tissue sarcoma.
• Previous receipt of at least one line of systemic anti-tumor therapy with subsequent disease progression.
• Presence of measurable target lesions according to RECIST 1.1 criteria.
• Sufficient biopsy tissue can be obtained from either metastatic or primary lesions.
• ECOG performance status score of 0 to 1.
• Expected overall survival ≥ 6 months.
• Adequate bone marrow reserve: HB ≥ 90 g/L; ANC ≥ 1.5×10⁹/L; PLT ≥ 80×10⁹/L.
• Adequate hepatic and renal function: total bilirubin ≤ 1.5 × upper limit of normal (ULN); ALT and AST ≤ 2.5 × ULN; serum creatinine ≤ ULN.
• Voluntary participation in the study with signed written informed consent form (ICF), good compliance, and willingness to comply with follow-up procedures.

Критерии исключения

• Patients with peripheral nervous system disorders caused by the disease, or a history of significant psychiatric or central nervous system disorders.
• Patients with severe infection or active peptic ulcer requiring treatment.
• Patients currently participating in other clinical trials or who have participated within the past 3 weeks.
• Patients deemed ineligible by the investigator for any other reason.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 1 центр

• Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of M — Шанхай

Идентификаторы

NCT: NCT07528079 · OSCAR-P

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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